<insert full date>
<insert Doc.Ref.>[only for CHMP reports]
Committee for Medicinal Products for Human Use (CHMP)
<Joint (Co)Rapporteurs Assessment Report<CHMP Assessment Report / <CHMP List of Questions> for <Product on derogations applicable to similar orphan products
International Nonproprietary Name: <INN
Procedure No. EMEA/H/C/XXX
Co-Rapporteur:
Table of contents
1. Introduction
2. <Clinical superiority assessment>
2.1. Efficacy
2.2. Safety
2.3. Major contribution to diagnosis or patient care
3. <Assessment on the inability of <applicant/MAH> to supply sufficient quantities of the <name of the authorised orphan product>
3.1. Outline of supply shortage of the <name of the authorised orphan product>
3.2. Demand and supply of the <name of the authorised orphan> in the EEA
3.3. Current treatment situation for the orphan condition
4. Conclusion
4.1. <Conclusion on clinical superiority>
4.2. <Conclusion on the inability of <applicant/MAH> to supply sufficient quantities of the <name of the authorised orphan product>
4.2.1. <List of questions>
5. <Assessment on the responses to the list of questions>
6. <Final conclusion >
7. Attachments
Administrative information
Type of information / NamesName of the medicinal product:
INN/Common name:
Applicant:
Applied therapeutic indication(s):
Pharmaco-therapeutic group
(ATC Code):
Pharmaceutical form(s) and strength(s):
Rapporteur’s contact person:
Co-Rapporteur’s contact person:
EMA Product Team Leader:
Names of the Rapporteur´s assessors:
Names of the Co-Rapporteur´s assessors:
1. Introduction
On date the <rapporteursCHMP concluded that the medicinal products <name of product> (INN of the product) and <name of the authorised orphan product(INN of authorised product) are similar as defined in Art.3 of the Commission Regulation (EC) No 847/2000 (attachment 1). Furthermore,Regulation (EC) No 141/2000 states:
- Article 8(1)
"Where a marketing authorisation in respect of an orphan medicinal product is granted (…), the Union and the Member States shall not, for a period of 10 years, accept another application for a marketing authorisation, or grant a marketing authorisation or accept an application to extend an existing marketing authorisation, for the same therapeutic indication, in respect of a similar medicinal product..." unless one of the three derogation as laid down in Art.8, paragraph 3 applies.
- Article 8(3):
By way of derogation from paragraph 1, and without prejudice to intellectual property law or any other provision of Community law, a marketing authorisation may be granted, for the same therapeutic indication, to a similar medicinal product if:
1.the holder of the marketing authorisation for the original orphan medicinal product has given his consent to the second applicant, or
2.the holder of the marketing authorisation for the original orphan medicinal product is unable to supply sufficient quantities of the medicinal product, or
3.the second applicant can establish in the application that the second medicinal product, although similar to the orphan medicinal product already authorised, is safer, more effective or otherwise clinically superior.
A marketing authorisation< An extension of the marketing authorisation<An authorisation>for <name of product> can therefore not be granted with the currently proposed indication, unless one of the above derogations are fulfilled.
In line with the above mentioned legislation, the applicant provided a derogation report:
The applicant> <MAH> provided a report justifying that their initial marketing application of> <extension application of <extension of indication of<name of product> is clinically superior to the authorised orphan medicinal product ,name of the authorised orphan product.
The <applicant> <MAH> provided a report that the holder of the marketing authorisation for the original orphan medicinal product is unable to supply sufficient quantities of the medicinal product.
This report reflects the <rapporteursCHMP assessment of the report on the claimed derogations.
2. Clinical superiority assessment
Consideration should be given to applicant’s arguments for claiming superiority; i.e. if these are based on theoretical assumptions, or are head-to-head (non-)clinical comparisons available. The design, conduct, reproducibility and results of such comparative studies or tests are to be evaluated. If no comparison data showing superiority, or justification for the lack of such data are presented, the applicant should be asked to address this in a response to the List of Questions.
2.1. Efficacy
Applicant’s position
<Rapporteurs’CHMP position
2.2. Safety
Applicant’s position
<Rapporteurs’CHMP position
2.3. Major contribution to diagnosis or patient care
Applicant’s position
<Rapporteurs’CHMP position
3. Assessment on the inability of<applicant/MAHto supply sufficient quantities of the name of the authorised orphan product>
3.1. Outline of supply shortage of the <name of the authorisedorphan product
Applicant’s position
<Rapporteurs’CHMP position
[This will be a question to be addressed by the applicant/MAH of the authorised orphan product.
Points to consider:
supply and availability of the product to the patients in the EU, local/national situations, declaration of the real causes of supply problem, supply issues caused by other reasons than manufacturing shortage (e.g. distributors, national legislation, re-imbursement) should be excluded, nature and extent of the problem, consequences for the applicant/MAH, manufacturing or other changes/rectifications conducted or planned, regulatory actions, revocation and suspension of MA, etc…]
3.2. Demand and supply of the name of the authorised orphan in the EEA
Applicant’s position
<Rapporteurs’CHMP position
[This will be a question to be addressed to the applicant/MAH of the authorised orphan product.
Points to consider: response to the current demand for the orphan product in the EEA, sales, orders and supply numbers, compassionate use, on-going clinical trials, planned batch releases and supply strategy in the EEA countries, duration of shortage, etc., in case of shortage, its duration, extent and cause.]
3.3. Current treatment situation for the orphan condition
Applicant’s position
<Rapporteurs’CHMP position
[These questions are to be addressed to patients’ and physicians’ organisations; depending on the disease, they might also be addressed to the member states (e.g. for questions related to registries).]
- How many patients within your remit do no receive < orphan medicinal product> as prescribed.
- If patients are not treated/sub-treated, is this due to an inability of <applicant/MAH> to supply <orphan product>
Points to consider: Numbers of patients currently treated with the orphan product with a full dose, with an adjusted dose or frequency of dosing, availability of the orphan product under different system (compassionate use), previous experience with orphan product shortage in this or similar indication, experience with “switching” patients to other treatment, etc…]
4. Conclusion
4.1. Conclusion on clinical superiority
4.2. Conclusion on the inability of <applicant/MAHto supply sufficient quantities of the <name of the authorised orphan product
4.2.1. List of questions
5. Assessment on the responses to the list of questions
6. Final conclusion
7. Attachments
- CHMP assessment report for <name of the product> on similarity with <name of the authorised orphan product.
<Joint (Co)Rapporteurs Assessment Report<CHMP Assessment Report> / <CHMP List of Questions> for <Product> on derogations applicable to similar orphan products
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